The Trump administration announced Monday that research supports leucovorin as a treatment for autism, with President Trump declaring "I think we found an answer to autism." The announcement centers on early medical trials of the decades-old, low-cost drug traditionally used to ease cancer treatment side effects, which have shown promise in improving communication in children with autism.
Clinical Evidence and Research Findings
Dr. Richard Frye, an American behavioral child neurologist and co-founder of the Autism Discovery and Treatment Foundation, conducted a pivotal 2018 study examining leucovorin's potential. The research, published in Nature journal Molecular Psychiatry, recruited 48 children with autism, giving approximately half folinic acid daily for 12 weeks while the other half received a placebo.
The study found "medium-to-large effect size" improvements in verbal communication abilities among those taking the drug. The therapeutic effect was particularly pronounced in children who had the folic acid-blocking genetic condition, supporting the underlying scientific rationale for the treatment.
Dr. Frye co-authored a 2021 review examining all available studies on the medication, analyzing 21 research papers. The comprehensive review concluded that leucovorin led to "significant improvements" in communication abilities across multiple studies.
Mechanism of Action and Scientific Rationale
Scientists first considered leucovorin for autism treatment after discovering that some people with autism spectrum disorder have a physiological defect in how their bodies process folate. Folate is a vitamin crucial to healthy brain development, which explains why many women take folic acid supplements during pregnancy.
Some individuals with autism have a condition where folate in the body is blocked from reaching the brain due to issues with cellular transmitters. These patients maintain normal folate levels in their system but lack adequate levels in the brain where it is needed. The theory suggests that folinic acid, or leucovorin, can correct this deficiency by delivering folate to the brain through alternative, unaffected channels.
Ongoing Research and Future Studies
Dr. Frye is currently conducting a larger study to better evaluate leucovorin's effectiveness on social and language deficits in autistic children. The expanded research involves 80 patients, with half receiving the drug twice daily for 12 weeks. Researchers are monitoring and assessing communication, social, and language skills through standardized questionnaires.
Results are expected next summer, with researchers hypothesizing that leucovorin "will significantly improve social communication as well as core and associated behavioral symptoms of ASD, and be well-tolerated with no significant adverse effects."
Drug Accessibility and Safety Profile
Leucovorin, also known as folinic acid, has an established safety profile in humans and is already administered to cancer patients to alleviate chemotherapy side effects. The medication is both cheap and easily accessible, with a pack of 12 pills costing approximately $30. It is approved in the US and UK as a cancer medication, though not yet for autism treatment applications.
Expert Cautions and Scientific Limitations
Medical experts emphasize significant limitations in the current evidence base. The research needed to establish leucovorin as a frontline autism treatment has not yet been fully funded or published. While the evidence shows promise, experts warn that leucovorin will not serve as a "silver bullet" for autism treatment.
The current scientific understanding of autism causes and symptom improvement remains "murky and extremely complicated," according to researchers. However, the backing of the administration could lead to a best-case scenario where a subset of patients gains access to a drug that can improve some symptoms in the near future.
Controversial Acetaminophen Claims
The Trump administration also plans to release statements suggesting that pregnant women taking acetaminophen, the main active ingredient in Tylenol, could face higher risks of having children with autism. This announcement has caused Kenvue, Tylenol's manufacturer, to experience significant stock declines.
A company spokesperson responded: "We believe independent, sound science clearly shows that taking acetaminophen does not cause autism." Former FDA Commissioner Scott Gottlieb stated Monday that there is no clear evidence that Tylenol causes autism and emphasized the need for further studies.
Clinical Success Stories
Individual cases have provided encouraging anecdotal evidence, including Mason Connor, a non-verbal three-year-old boy from Arizona who spoke for the first time just days after starting folinic acid medication in one of Dr. Frye's trials. Such cases highlight the potential impact for families dealing with autism spectrum disorder, though they represent individual responses rather than population-wide efficacy data.
